3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
4
Rare Diseases
across 9 areas
0
News (30d)
Quiet
USC-CHLA Institute for Pediatric Clinical Research is a company with 3 orphan drug designations across 4 rare diseases.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| neuroblastoma | Fenretinide | Des.TrialAppr. |
| pediatric hepatocellular carcinoma | Buthionine sulfoxamine | Des.TrialAppr. |
| pediatric ovarian dysgerminoma | Buthionine sulfoxamine | Des.TrialAppr. |
| pediatric-onset Graves disease | Buthionine sulfoxamine | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
77
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
77
overlap in 2+ diseases
0/4
candidate diseases
0
avg importance: 0
0
affecting portfolio